GSK Phase III Exon Skipping Trial in the States This Fall?

I received this forward a couple weeks ago from our genetic counselor at the U of MN. It was written to her from the University's Clinical Research Coordinator.

Hello,

I spoke with Marlene Jordan this afternoon regarding the GSK exon skipping study. Right now the timeline consists of us getting the final protocol in June, investigator’s meeting in August with the hope to screen the first subjects in September (dependent on site IRB and contract approval).

She said this information is okay to share with families.

Marlene also mentioned a glitch on the clinicaltrials.gov website regarding the list of potential sites. They hope to get this fixed soon, but assured me we are a site for the study.

Replies to This Discussion

As you are aware, GSK is working with FDA to provide the information they have requested with the intent of having the partial clinical hold lifted. We are on track to receive a reply from the FDA next month. Assuming the partial hold is lifted at that time, the study will proceed forward with the investigator meeting in August and enrollment will begin in late Aug/early September.

As a reminder, our study design is a 6 month treatment period & 6 month post-treatment period. We plan to offer all subjects enrollment into an open-label extension study at the conclusion of the 114876 study.

I'm not sure, but my understanding is the IND was filed and the FDA asked for further information. That information was provided and GSK is now expecting a reply from the FDA next month. If all goes accordingly they should be on track to begin US trials this fall.

I'll ask if there's any more information on this.

Mindy said:

Can you ask them when they expect the IND to be opened? Has it been filed yet?